After U.S. markets closed yesterday afternoon, Ultragenyx Pharmaceutical Inc. (RARE:NASDAQ), a biopharmaceutical company engaged in the development of novel products for rare and ultra-rare diseases, announced "positive topline safety and efficacy data from Cohort 3 and longer-term data from Cohort 2 of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency."
The company advised that the objective of the Phase 1/2 study of DTX301 is to "evaluate the change in the rate of ureagenesis, ammonia levels, neurocognitive assessment, biomarkers, and safety of DTX301 in patients with OTC deficiency." The firm explained that OTC deficiency is the most common urea cycle disorder that affects more than 10,000 individuals worldwide caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. The company indicated that to date there have been no infusion-related adverse events and no treatment-related serious adverse events reported in the study.
Ultragenyx indicated that DTX301 has been granted Orphan Drug Designation in the U.S. and Europe and is "an investigational AAV type 8 gene therapy designed to deliver stable expression and activity of OTC following a single intravenous infusion, which has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism."
Eric Crombez, M.D., chief medical officer of the Ultragenyx Gene Therapy development unit, commented, "We are encouraged to see a more uniform response at the higher doses including three female responders. To date, three patients in the study have discontinued alternate pathway medication and liberalized their diets while remaining clinically and metabolically stable...We are moving to prophylactic steroid use in the next cohort as we believe this could further enhance the level and consistency of expression that we have demonstrated so far.".
Ultragenyx is headquartered in Novato, Calif., and is a biopharmaceutical company specializing in developing novel products for the treatment of serious rare and ultra-rare genetic diseases. The company aims at addressing diseases with high unmet medical need with clear biology for treatment for which there are no presently approved therapies for treating the underlying disease.
Ultragenyx Pharmaceutical started the day with a market capitalization of approximately $2.5 billion with about 57.77 million outstanding shares and a short interest of around 10.6%. RARE shares opened nearly 12% higher today at $48.91 (+$5.18, +11.85%) over yesterday's $43.73 closing price. The stock has traded today between $44.84 and $58.54 per share and is currently trading at $58.44 (+$14.71, +33.64%).
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